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At St. Jude, doctors and scientists work to improve childhood cancer survival rates as well as enhance patients’ long-term quality of life after treatment

Memphis, Tennessee, September 2, 2013

Despite a steady rise in survival rates, childhood cancer remains the leading cause of death by disease among young Americans. St. Jude Children’s Research Hospital doctors and scientists are working to change that statistic.

September marks Childhood Cancer Awareness Month, a time to highlight efforts to reduce the disease’s toll on children. At St. Jude, the past year has brought advances in understanding and treating childhood cancer, particularly in the areas of genomics and survivorship.

“One child dying of cancer is one too many and the need to find better treatments is as critical as ever,” said Dr. William E. Evans, St. Jude director and CEO. “As we learn more and more about the genetic causes of pediatric cancer through genomic research, treating these cancers becomes more complex and challenging.”

Genomic studies have shown researchers that diseases previously thought to be single entities actually include multiple subtypes that often don’t respond to standard treatment. The challenge now is determining the genetic missteps that cause these cancers and finding existing drugs or developing new drugs for targeted treatment.

A number of significant discoveries related to these efforts have been made through the Pediatric Cancer Genome Project, a collaboration between St. Jude and Washington University to map the complete normal and cancer genomes of 700 young patients. This effort to understand what drives some of the most aggressive childhood cancers is the largest project of its type. When project data was made available in May 2012, it marked the largest-ever release of comprehensive human cancer genome data for free access by the global scientific community. The amount of information shared more than doubled the volume of high-coverage, whole genome data available from all human genome sources combined.

“The sequence data represent billions of clues, and the real detective work for researchers is figuring out what they mean for the bigger picture,” Evans said. “Ultimately, this information provides the foundation for the clinical trials that lead to more effective therapies. At St. Jude, translating our basic research to clinical applications is a major focus and is why we currently develop more clinical trials than any other children’s hospital.”

Pediatric Cancer Genome Project discoveries during the past year include identifying missteps in two genes responsible for more than 50 percent of diffuse low-grade gliomas, hard-to-treat tumors of the brain and spine. Researchers also uncovered a genetic mistake responsible for almost 30 percent of cases of acute megakaryoblastic leukemia, an uncommon subtype of childhood leukemia with a poor prognosis, which paves the way for needed treatment advances.

Today’s cancer researchers also have to be concerned with enhancing the quality of life for children when treatment ends.

“With more childhood cancer patients surviving into adulthood, there is a growing need to study the late health effects of live-saving treatments,” Evans said.

This year, St. Jude published results from a one-of-a-kind study that included complete medical assessments of more than 1,700 adults who were treated at St. Jude as children. Researchers found the adult childhood cancer survivors had a significant amount of undiagnosed, serious disease as adults. The risks varied by the treatment the individual received.

The study had dramatic benefits for many of the participants, as the screenings identified previously undiagnosed conditions, such as breast cancer and heart problems. Insights like these showcase the importance for survivors to share their medical histories with their current physicians to monitor them for risks. The findings also help today’s researchers devise strategies to avoid the side effects of treatment now and in the future.